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molecule","productStatus":"Approved","date":"October 2022","year":"2022","type":"Not Applicable","leadProduct":"Ravulizumab","moa":"Complement 5a receptor","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Alexion Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Phase III","highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Alexion Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Alexion Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alexion Aims To Advance NMOSD Treatment Landscape With Exceptional ULTOMIRIS (ravulizumab-cwvz) Efficacy Data At ECTRIMS 2022","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"","productType":"Large molecule","productStatus":"Approved","date":"October 2022","year":"2022","type":"Not Applicable","leadProduct":"Ravulizumab","moa":"Complement 5a receptor","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Alexion Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Phase III","highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Alexion Pharmaceuticals \/ AstraZeneca","highestDevelopmentStatusID":"10","companyTruncated":"Alexion Pharmaceuticals \/ AstraZeneca"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alexion Aims To Advance NMOSD Treatment Landscape With Exceptional Ultomiris Efficacy Data At ECTRIMS 2022","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"","productType":"Large molecule","productStatus":"Approved","date":"October 2022","year":"2022","type":"Not Applicable","leadProduct":"Ravulizumab","moa":"Complement 5a receptor","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Alexion Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Phase III","highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Alexion Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Alexion Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"LogicBio Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Alexion, AstraZeneca Rare Disease, Completes Acquisition of LogicBio\u00ae Therapeutics","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2022","year":"2022","type":"Acquisition","leadProduct":"AAV-based Gene Therapy","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Alexion Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Alexion Pharmaceuticals \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"2","companyTruncated":"Alexion Pharmaceuticals \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Neurimmune","pharmaFlowCategory":"D","amount":"$760.0 million","upfrontCash":"$30.0 million","newsHeadline":"AstraZeneca and Neurimmune Sign Exclusive Global Collaboration and Licence Agreement to Develop and Commercialise NI006","therapeuticArea":"Cardiology\/Vascular Diseases","highestDevelopmentStatus":"Phase I","country":"SWITZERLAND","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2022","year":"2022","type":"Collaboration","leadProduct":"NI006","moa":"Transthyretin amyloid","url1":"","url2":"","graph1":"Cardiology\/Vascular Diseases","graph2":"Phase I","graph3":"Alexion Pharmaceuticals","amount2":0.76000000000000001,"therapeuticAreaNew":"Cardiology\/Vascular Diseases","highestDevelopmentStatusNew":"Phase I","highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Cardiovascular Diseases","productSubType":"","amount2New":0.76000000000000001,"dosageForm":"Infusion","sponsorNew":"Alexion Pharmaceuticals \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"6","companyTruncated":"Alexion Pharmaceuticals \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"JCR Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"JCR Pharmaceuticals Announces Research Collaboration, Option and License Agreement with Alexion to Develop a Therapy Using J-Brain Cargo\u24c7 for Neurodegenerative Disease","therapeuticArea":"Neurology","highestDevelopmentStatus":"Discovery","country":"","productType":"Undisclosed","productStatus":"New Molecular Entity","date":"April 2023","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Neurology","graph2":"Discovery","graph3":"Alexion 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2023","year":"2023","type":"Not Applicable","leadProduct":"Danicopan","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Alexion Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Approved","highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","productSubType":"Small molecule","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Alexion Pharmaceuticals \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"12","companyTruncated":"Alexion Pharmaceuticals \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"BridgeBio Pharma","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AG10 (Acoramidis) is an Investigational, Orally-Administered Small Molecule Designed to Potently Stabilize Tetrameric Transthyretin (TTR)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2023","year":"2023","type":"Not Applicable","leadProduct":"Acoramidis Hydrochloride","moa":"","url1":"","url2":"","graph1":"Cardiology\/Vascular Diseases","graph2":"Phase III","graph3":"Alexion Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Cardiology\/Vascular Diseases","highestDevelopmentStatusNew":"Phase III","highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Cardiovascular Diseases","productSubType":"","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Alexion Pharmaceuticals \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"Alexion Pharmaceuticals \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Pfizer Inc","pharmaFlowCategory":"D","amount":"$1,000.0 million","upfrontCash":"Undisclosed","newsHeadline":"Alexion, AstraZeneca Rare Disease, Enters Agreement with Pfizer to Acquire a Portfolio of Preclinical Rare Disease Gene Therapies","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2023","year":"2023","type":"Licensing Agreement","leadProduct":"Gene Therapy","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Alexion Pharmaceuticals","amount2":1,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Preclinical","highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":1,"dosageForm":"","sponsorNew":"Alexion Pharmaceuticals \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Alexion Pharmaceuticals \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Verge Genomics","pharmaFlowCategory":"D","amount":"$882.0 million","upfrontCash":"$42.0 million","newsHeadline":"Verge Genomics Announces Artificial Intelligence-Enabled Drug Discovery Collaboration with Alexion for Rare Neurodegenerative and Neuromuscular Diseases","therapeuticArea":"Neurology","highestDevelopmentStatus":"Discovery","country":"","productType":"Undisclosed","productStatus":"New Molecular Entity","date":"September 2023","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Neurology","graph2":"Discovery Platform","graph3":"Alexion Pharmaceuticals","amount2":0.88,"therapeuticAreaNew":"Neurology","highestDevelopmentStatusNew":"Discovery Platform","highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Neurology","productSubType":"","amount2New":0.88,"dosageForm":"","sponsorNew":"Alexion Pharmaceuticals \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"3","companyTruncated":"Alexion Pharmaceuticals \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Pfizer Inc","pharmaFlowCategory":"D","amount":"$1,000.0 million","upfrontCash":"Undisclosed","newsHeadline":"Alexion Completes Purchase and Licence Agreement for Early-Stage Rare Disease Gene Therapy Portfolio from Pfizer","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2023","year":"2023","type":"Licensing Agreement","leadProduct":"Gene Therapy","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Alexion Pharmaceuticals","amount2":1,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Preclinical","highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":1,"dosageForm":"","sponsorNew":"Alexion Pharmaceuticals \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Alexion Pharmaceuticals \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"JCR Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"JCR Pharmaceuticals Announces Achievement of Milestone Using J-Brain Cargo\u00ae Technology for Neurodegenerative Disease in Research Collaboration with Alexion","therapeuticArea":"Neurology","highestDevelopmentStatus":"Discovery","country":"","productType":"Undisclosed","productStatus":"New Molecular Entity","date":"March 2024","year":"2024","type":"Collaboration","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Neurology","graph2":"Discovery","graph3":"Alexion Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Neurology","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Neurology","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Alexion Pharmaceuticals \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"2","companyTruncated":"Alexion Pharmaceuticals \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ULTOMIRIS\u00ae (ravulizumab-cwvz) Approved in the US for the Treatment of Adults with Neuromyelitis Optica Spectrum Disorder (NMOSD)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"","productType":"Large molecule","productStatus":"Approved","date":"March 2024","year":"2024","type":"Not Applicable","leadProduct":"Ravulizumab","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Alexion Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Approved","highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Alexion Pharmaceuticals \/ AstraZeneca","highestDevelopmentStatusID":"12","companyTruncated":"Alexion Pharmaceuticals \/ AstraZeneca"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"BridgeBio Pharma","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BridgeBio Pharma Shares Positive Results of Single-Arm Phase 3 Study of Acoramidis in Japanese Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Including No Mortality Reported in the Trial at 30 Months","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2024","year":"2024","type":"Not Applicable","leadProduct":"Acoramidis Hydrochloride","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alexion Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase III","highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Alexion Pharmaceuticals \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"Alexion Pharmaceuticals \/ Alexion Pharmaceuticals"}]

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          Luxepack
                          Not Confirmed
                          Luxepack
                          Not Confirmed

                          Details : Ultomiris (ravulizumab) has been approved in the US as the first and only long-acting C5 complement inhibitor for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder.

                          Brand Name : Ultomiris

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          March 25, 2024

                          Lead Product(s) : Ravulizumab

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Sponsor : AstraZeneca

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          02

                          Luxepack
                          Not Confirmed
                          Luxepack
                          Not Confirmed

                          Details : The collaboration aims to develop an undisclosed initial therapeutic molecule that applies JCR’s proprietary J-Brain Cargo®, blood-brain barrier penetration technology, for the treatment of a neurodegenerative disease.

                          Brand Name : Undisclosed

                          Molecule Type : Undisclosed

                          Upfront Cash : Undisclosed

                          March 19, 2024

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Neurology

                          Highest Development Status : Discovery

                          Recipient : JCR Pharmaceuticals

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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                          03

                          Luxepack
                          Not Confirmed
                          Luxepack
                          Not Confirmed

                          Details : AG-10 (acoramidis) is an investigational, next-generation, orally-administered, highly potent, small molecule stabiliser of transthyretin (TTR). It is being evaluated in phase 3 clinical trials for the treatment of Transthyretin Amyloid Cardiomyopathy.

                          Brand Name : AG10

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          February 02, 2024

                          Lead Product(s) : Acoramidis Hydrochloride

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Recipient : BridgeBio Pharma

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          04

                          Luxepack
                          Not Confirmed
                          Luxepack
                          Not Confirmed

                          Details : Under the agreement, Alexion expand its rare disease gene therapy portfolio by gaining Pfizer’s early-stage preclinical gene therapy programmes. The agreement furthers Alexion's commitment to advancing next-generation genomic medicines.

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          September 20, 2023

                          Lead Product(s) : Gene Therapy

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Preclinical

                          Recipient : Pfizer Inc

                          Deal Size : $1,000.0 million

                          Deal Type : Licensing Agreement

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                          05

                          Luxepack
                          Not Confirmed
                          Luxepack
                          Not Confirmed

                          Details : The collaboration aims to identify novel drug targets for rare neurodegenerative and neuromuscular diseases by leveraging CONVERGE, a full-stack platform that combines highly predictive human tissue datasets with machine learning to find new targets.

                          Brand Name : Undisclosed

                          Molecule Type : Undisclosed

                          Upfront Cash : $42.0 million

                          September 08, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Neurology

                          Highest Development Status : Discovery

                          Recipient : Verge Genomics

                          Deal Size : $882.0 million

                          Deal Type : Collaboration

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                          06

                          Luxepack
                          Not Confirmed
                          Luxepack
                          Not Confirmed

                          Details : Under the agreement, Alexion will expand its rare disease gene therapy portfolio by gaining Pfizer’s early-stage preclinical gene therapy programmes. The agreement furthers Alexion's commitment to advancing next-generation genomic medicines.

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          July 28, 2023

                          Lead Product(s) : Gene Therapy

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Preclinical

                          Recipient : Pfizer Inc

                          Deal Size : $1,000.0 million

                          Deal Type : Licensing Agreement

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                          07

                          Luxepack
                          Not Confirmed
                          Luxepack
                          Not Confirmed

                          Details : AG10 (acoramidis) is an investigational, orally-administered small molecule designed to potently stabilize tetrameric transthyretin (TTR), being investigated for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM).

                          Brand Name : AG10

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          July 17, 2023

                          Lead Product(s) : Acoramidis Hydrochloride

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Recipient : BridgeBio Pharma

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          08

                          Luxepack
                          Not Confirmed
                          Luxepack
                          Not Confirmed

                          Details : ALXN2040 (danicopan) is an investigational oral medicine in development as an add-on to standard of care C5 inhibitor therapy Ultomiris (ravulizumab) or Soliris (eculizumab) for patients with PNH who experience clinically significant EVH.

                          Brand Name : ALXN2040

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          June 09, 2023

                          Lead Product(s) : Danicopan,Eculizumab,Ravulizumab

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Recipient : AstraZeneca

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          09

                          Luxepack
                          Not Confirmed
                          Luxepack
                          Not Confirmed

                          Details : Under the agreement, the two companies will begin research and development of new treatments using the J-Brain Cargo®, blood-brain barrier (“BBB”) penetration technology, in combination with an undisclosed effector molecule for the treatment of a ne...

                          Brand Name : Undisclosed

                          Molecule Type : Undisclosed

                          Upfront Cash : Undisclosed

                          April 03, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Neurology

                          Highest Development Status : Discovery

                          Recipient : JCR Pharmaceuticals

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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                          10

                          Luxepack
                          Not Confirmed
                          Luxepack
                          Not Confirmed

                          Details : The acquisition creates an opportunity to accelerate Alexion’s growth in genomic medicines through unique technology, an experienced rare disease R&D team, and expertise in pre-clinical development.

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          November 16, 2022

                          Lead Product(s) : AAV-based Gene Therapy

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Discovery

                          Recipient : LogicBio Therapeutics

                          Deal Size : Undisclosed

                          Deal Type : Acquisition

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